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NCT04657094

Acalabrutinib for the Treatment of Relapsed or Refractory Autoimmune Hemolytic Anemia in Patients With Chronic Lymphocytic Leukemia

Active, enrolled Phase 2 Results posted Last updated 27 April 2025
What this trial tests

Phase 2 trial testing Acalabrutinib in Autoimmune Hemolytic Anemia in 4 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
16 March 2021
Primary endpoint
1 April 2023
27 February 2026

Quick facts

Lead sponsorCity of Hope Medical Center
PhasePhase 2
StatusActive, enrolled
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment4
Start date16 March 2021
Primary completion1 April 2023
Estimated completion27 February 2026
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

City of Hope Medical Center

Who can join

18 and older, any sex, with Autoimmune Hemolytic Anemia or Chronic Lymphocytic Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Autoimmune Hemolytic Anemia (AIHA) - Overall Response Rate (ORR) Primary · Participants were assessed at the end of the 6-week therapy.

ORR is defined as proportion of patients who achieve complete response (CR) and partial response (PR). The probability of having AIHA-ORR at 6 cycles were measured and reported with 95% exact confidence interval (CI). An exact binomial test against a null hypothesis of 30% rate was performed at the 1-sided alpha of 0.05 to determine whether the AIHA-ORR rate at 6 cycles is disappointing or promising.

GroupValue95% CI
Treatment (Acalabrutinib)679 – 99

Adverse events — posted to ClinicalTrials.gov

Time frame: From the initial study treatment up to 12 months after the end of the treatment, approximately two years.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment (Acalabrutinib)
Serious: 2/4 (50%)
Deaths: 0/4

Serious adverse events (2 terms)

ReactionSystemTreatment (Acalabrutinib)
DRESS SyndromeImmune system disorders
COVID-19Infections and infestations
Other adverse events (40 terms — click to expand)

ReactionSystemTreatment (Acalabrutinib)
FatigueGeneral disorders
FallInjury, poisoning and procedural complications
Alanine aminotransferase increasedInvestigations
Aspartate aminotransferase increasedInvestigations
Lymphocyte count decreasedInvestigations
White blood cell decreasedInvestigations
HyperglycemiaMetabolism and nutrition disorders
HypocalcemiaMetabolism and nutrition disorders
HypoglycemiaMetabolism and nutrition disorders
HyponatremiaMetabolism and nutrition disorders
ArthralgiaMusculoskeletal and connective tissue disorders
Dry skinSkin and subcutaneous tissue disorders
Rash maculo-papularSkin and subcutaneous tissue disorders
AnemiaBlood and lymphatic system disorders
Abdominal painGastrointestinal disorders
Dental FracturesGastrointestinal disorders
DiarrheaGastrointestinal disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
Mucositis oralGastrointestinal disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
Edema limbsGeneral disorders
Neck NumbnessGeneral disorders
Non-cardiac chest painGeneral disorders
DRESS SyndromeImmune system disorders
Skin infectionInfections and infestations
BruisingInjury, poisoning and procedural complications
Blood lactate dehydrogenase increasedInvestigations
INR increasedInvestigations
Lymphocyte count increasedInvestigations
Neutrophil count decreasedInvestigations
Platelet count decreasedInvestigations
AnorexiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
HeadacheNervous system disorders
DeliriumPsychiatric disorders
CoughRespiratory, thoracic and mediastinal disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
Nasal congestionRespiratory, thoracic and mediastinal disorders
HyperhidrosisSkin and subcutaneous tissue disorders

Most-reported serious reactions: DRESS Syndrome, COVID-19.

Data from ClinicalTrials.gov NCT04657094 adverse events section.

Sponsor's own description

This phase II trial studies the effect of acalabrutinib in treating autoimmune hemolytic anemia that has come back (relapsed) or has not responded to previous treatment (refractory) in patients with chronic lymphocytic leukemia. Acalabrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Bruton's Kinase Inhibitors for the Treatment of Immunological Diseases: Current Status and Perspectives.
    Robak E, Robak T. · · 2022 · cited 45× · PMID 35628931 · DOI 10.3390/jcm11102807
  2. BTK inhibitors: past, present, and future.
    Cool A, Nong T, Montoya S, Taylor J. · · 2024 · cited 37× · PMID 39025681 · DOI 10.1016/j.tips.2024.06.006
  3. Clinical Trials of the BTK Inhibitors Ibrutinib and Acalabrutinib in Human Diseases Beyond B Cell Malignancies.
    Zhu S, Jung J, Victor E, Arceo J, et al · · 2021 · cited 23× · PMID 34778053 · DOI 10.3389/fonc.2021.737943
  4. [The treatment strategies of autoimmune hemolytic anemia].
    Yue WR, Wu T, Wang XQ. · · 2024 · cited 1× · PMID 39134500 · DOI 10.3760/cma.j.cn121090-20231027-00236
  5. [Efficacy and safety of zanubrutinib in treating refractory/relapsed warm autoimmune hemolytic anemia].
    Shen SP, Yang C, Chen M, Han B. · · 2025 · PMID 40425458 · DOI 10.3760/cma.j.cn121090-20241110-00442
  6. [How I diagnose and treat autoimmune hemolytic anemia].
    Fu R, Yu H. · · 2022 · PMID 36709181 · DOI 10.3760/cma.j.issn.0253-2727.2022.11.005

Verify or expand the search:

Other trials of Acalabrutinib

Trials testing the same drug.

Other recruiting trials for Autoimmune Hemolytic Anemia

Currently open trials in the same condition.

Other City of Hope Medical Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04657094.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing